Clinical Trials: Improving the Lives of People with Diabetes
By April Hopcroft
At a virtual panel, experts discussed the benefits and challenges of participating in clinical trials, with a focus on recruiting participants from diverse backgrounds. Panelists explored diabetes-related eye disease and beta cell replacement as key areas of clinical trial research.
Over the past century, diabetes care has improved dramatically, thanks to key inventions: the discovery of insulin, the creation of continuous glucose monitors (CGMs), and the development of SGLT-2 inhibitors and GLP-1 agonists to protect the kidney and the heart, to name a few.
Yet we wouldn’t be where we are today without clinical trials and people with diabetes who participated in these studies.
While clinical trials are a key part of advancing diabetes care, not all people with diabetes are aware of these studies or able to access them. During diaTribe’s panel discussion, “Clinical Trials in Diabetes: Working to Improve Lives,” experts discussed the steps involved in clinical research and strategies to make trials more accessible, especially to people of color and others from underrepresented backgrounds.
The panel was moderated by Dr. Alan Moses, Board Chair of the diaTribe Foundation. Delivering opening remarks, Dr. Julie Hsieh, Staff Fellow at the The Office of Minority Health and Health Equity, emphasized the FDA’s commitment to increasing enrollment of diverse populations in clinical trials through education and outreach to address barriers to participation. The panelists included:
-
Anastasia Albanese-O'Neill, Assistant Vice President of T1D Education and Clinical Implementation at JDRF International
-
Dr. Manuel Amador, Medical Director, US Medical Affairs in Ophthalmology at Genentech
-
Dr. Anne Peters, Professor of Clinical Medicine, Clinical Scholar, Keck School of Medicine of USC
-
Dr. Jennifer Sun, Associate Professor, Harvard Department of Ophthalmology
What are the phases of clinical trials?
In order for a new drug to be approved by the FDA, it must go through rigorous testing. Clinical trials usually involve four stages, three of which must be complete before the drug receives FDA approval.
-
Phase 1: These trials test the drug in a small group of people (about 20 or fewer) who don’t have underlying health conditions to determine safety and dosage.
-
Phase 2: These medium-sized studies (about 100 people) investigate how the treatment affects the body in people living with the health condition and whether it has any benefit.
-
Phase 3: These large trials test whether the new therapy is better compared to existing treatments and can include up to several thousands of participants.
-
Phase 4: After a drug is approved by the FDA, data is collected to understand its long-term safety and efficacy.
Joining clinical trials
From filling out a questionnaire about diabetes stigma to participating in an in-depth investigation of a novel treatment to delay type 1 diabetes, there are many different ways to be involved in diabetes research.
Panelists stressed the importance of gaining informed consent and educating participants before they decide to join a clinical trial. Informed consent is a founding principle of research ethics and aims to ensure that participants can enter studies with full knowledge of what the research involves, including potential harms and benefits.
“At the core of a person’s decision to participate in a clinical trial is informed consent and a clear understanding of how these studies work,” Albanese-O'Neill said, adding: “People should participate where they feel comfortable.”
Benefits of participating in clinical trials
In addition to getting the chance to test-drive new therapies or technologies and the opportunity to shape the future of diabetes care, there are other less obvious benefits of participating in research.
One important benefit is the opportunity to receive high quality care through regular contact and follow-up visits. Trial participants gain access to an extended support system of healthcare providers and study coordinators, extra reminders and follow-up appointments, as well as additional diabetes self-management education. This extra attention can translate to better health outcomes, Amador said.
Trials may involve many hours spent waiting at a clinic (such as while completing an oral glucose tolerance test), which can provide an opportunity for education.
“If you have a couple hours with a diabetes educator, you can really dig deep into diabetes education,” Albanese-O'Neill said. “[This provides] additional knowledge that is powerful and follows people after leaving the study.”
While the extra care and education isn’t necessarily intentional, Moses said, it’s something that is often skipped over in typical care outside of a clinical trial. “The support systems, the appointment reminders, the education are all things that unfortunately, clinicians, whether they be physicians or nurse educators, just don't have the time or opportunity to deliver in routine practice in a way that can really help individuals understand the challenges or the problems that they have.”
Another benefit of participating in research is the opportunity to develop close relationships with healthcare providers through frequent visits, especially for studies that last for many years. “One of the joys of being a trialist is developing long-term relationships with patients,” Sun, the professor at Harvard, said.
Lastly, there’s the feel-good benefit of helping to advance treatment for other people with diabetes. Albanese-O'Neill said that participating in research can also provide a sense of hope, as it’s an opportunity to do something to improve diabetes care.
Trust is key when recruiting trial participants
Peters discussed her work recruiting participants from the Eastside of Los Angeles, which is home to the largest Hispanic community in the US. Initially, she said many people were suspicious of clinical trials. They believed researchers just wanted to use them for data and had no desire to provide lasting care – certainly understandable given the history of medical racism in the US.
“I promised not to leave and that I would make everything community-focused,” Peters said. “So I asked the people in those communities what I can do to help them. In this way, we've developed trust. I know they will join us for clinical trials. They're pretty good at it, because they trust us."
In order to successfully enroll participants, Peters and her team have had to think creatively to address barriers such as transportation access or strict working hours. For instance, study coordinators relied on Ubers to pick up participants and selected clinics with evening and weekend hours. Hiring study coordinators who come from the Eastside, and therefore know the patients well and understand the difficulties they face, was also important.
Likewise, Sun said it’s really important to build a culture of understanding both on the medical side and for patients. Sun said she seeks to help patients “understand that their participation in clinical research trials has enormous value decades and years into the future for their families, loved ones, and other people with diabetes.”
For Peters, one of the challenges of clinical trials is that participants no longer have access to the technologies or therapies after finishing the study.
“When someone completes a diabetes technology trial, then I have to stop giving them access to the device, which helped them get their A1C down from 12% to 7%,” Peters said. “I can’t do that morally, so I get donors from my [wealthier] Westside practice to help fund ways for these patients to stay on the devices.”
Ensuring diversity of clinical trials
Panelists also touched on the continued importance of access and diversity in clinical trials, with a particular focus on research for diabetes-related eye disease. Treatments for diabetic retinopathy and diabetic macular edema (DME) have come a long way over the past decades. Access to these therapies isn’t guaranteed, however; many people across the world do not have ways to obtain the specialized care and technology required for treatment. Sun said that clinical trials will be crucial to close global gaps in access, as researchers are looking for new treatments that are readily available, affordable, and scalable.
Amador cited Genentech's Elevatum study, which is investigating the drug faricimab (approved under brand name Vabysmo) in underrepresented people with DME. This includes Black, Hispanic, Latin American, Native American, native Alaskan, or Native Hawaiian/Pacific Islander populations.
In designing this study, Genentech took several steps to address barriers that prevented people from these backgrounds from enrolling in trials.
-
The upper limit for A1C was 12%, rather than the usual cutoff of 10%. This is important because people from underrepresented backgrounds tend to start clinical trials with more severe disease and are often excluded from research due to their high A1C.
-
The trial includes healthcare providers from the same background as patients. Amador said that conversations between patients and providers are much easier if the provider shares the same language, skin color, or cultural background.
-
Trial resources were translated into multiple languages. For instance, while many studies translate information into Spanish to reach Latinos, Amador noted the importance of also translating into Portuguese, as about one-third of Latinos are from Brazil.
Designing clinical trials to include patient education and input
Over the past year, technologies such as Vertex Pharmaceuticals’ VX-880 stem cell replacement or the cell therapy Lantidra have shown potential to replace or restore beta cells in people with type 1 diabetes. While there is strong interest in enrolling in trials for these therapies, it’s important to make sure patients understand the complexity and commitment involved in participating, Albanese-O'Neill said.
Patient education is especially important for studies that require surgery or immunosuppression. Participants may need to take extended time off from work to recover from procedures, and those taking immunosuppressants will have a weakened immune system.
She also noted that some studies require participants to manage their diabetes differently than they’re accustomed to, so it’s important to know this before entering a study.
Trials for type 1 diabetes therapies may also require multiple appointments with different specialists and many labs and tests to monitor the treatments – in other words, significant time, effort, and cost.
Recognizing these challenges, JDRF has formed a participant advisory council to include the full community of people with type 1 diabetes in the earliest stages of clinical trial design. “The goal of this group is to create a collection of diverse voices of people who are underinsured, uninsured, or very well insured,” Albanese-O'Neill said.
“We want to include people of all races and ethnicities and ages and in different stages of type 1 diabetes, [from] early stage type 1 diabetes all the way through to having different complications.”
Learn more about clinical trials for diabetes:
